The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
May 14th 2024
Treatment with rozanolixizumab was safe among patients with CIDP, resulted in reduced immunoglobulin levels as expected, but did not have an impact on efficacy.
Improving Care for Patients with Motor Complications of Parkinson Disease: Role of Technology and Data
View More
Clinical Consultations™: Considerations for Customizing Care Plans for Patients with Parkinson Disease Psychosis
View More
Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
September 15, 2024
Register Now!
Mastering MS: Translating Evidence into Optimal Management Plans
View More
Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care
View More
Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes
View More
5th Annual International Congress on the Future of Neurology®
View More
Clinical Consultations™: Managing Depressive Episodes in Patients with Bipolar Disorder Type II
View More
Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
View More
Medical Crossfire®: Understanding the Advances in Bipolar Disease Treatment—A Comprehensive Look at Treatment Selection Strategies
View More
'REEL’ Time Patient Counseling: The Diagnostic and Treatment Journey for Patients With Bipolar Disorder Type II – From Primary to Specialty Care
View More
Burst CME: Optimizing Therapy in Parkinson’s Disease
View More
Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
View More
Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
View More
Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
View More
Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
View More
Addressing Healthcare Inequities: Bridging the Gap in Multiple Sclerosis – A Focus on Clinical and Healthcare Disparities in Black Patients
View More
Individualizing Treatment for Patients with Generalized Myasthenia Gravis
View More
Patient, Provider & Caregiver Connection™: Reducing the Burden of Parkinson Disease Psychosis with Personalized Management Plans
View More
Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
View More
Addressing the Core Features of Rett Syndrome With Trofinetide
March 29th 2019The study author, a pediatric neurologist at Baylor College of Medicine and director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, spoke about the investigational Rett syndrome treatment’s potential going forward.
Trofinetide Triumphs in Phase 2 Study of Rett Syndrome
March 28th 2019Of the 5 syndrome-specific efficacy measurements, trofinetide showed statistical benefits for patients with Rett syndrome on the Rett Syndrome Behavior Questionnaire, Clinical Global Impression Scale-Improvement, and the RTT Clinician Domain Specific Concerns-Visual Analog Scale.
Idebenone Reduces Respiratory Function Loss in Duchenne Muscular Dystrophy
March 22nd 2019SYROS study data showed that switching to and maintaining long-term treatment with idebenone was associated with a 50% reduction in the annual rate of decline in forced vital capacity percent of predicted.
Head of NCI Ned Sharpless Named Acting FDA Commissioner
March 13th 2019Ned Sharpless, MD, the director of the National Cancer Institute, has been named to the position of acting FDA Commissioner. The announcement was made just a week after the current commissioner, Scott Gottlieb, MD, announced his plans to resign in early April.
OnabotulinumtoxinA sBLAs for Upper, Lower Limb Spasticity Accepted by FDA
March 7th 2019The Allergan product was granted Priority Review for upper limb spasticity and is expected to be decided on in the second quarter of 2019, while the lower limb indication will have its evaluation by the fourth quarter.
Rare Neurologic Diseases Are Not Truly Rare
February 28th 2019Despite being labeled as rare diseases, a number of neurologic conditions impact more patients than most would believe. The consultant with expertise in ophthalmology, gene therapy, and rare and orphan diseases, chimed in about how these diseases can often be overlooked.
Addressing the Challenges in Determining Pediatric Brain Death
February 27th 2019The pediatric critical care medicine attending physician in the Department of Anesthesiology and Critical Care Medicine at the Children’s Hospital of Philadelphia spoke about how physicians can better address brain death in pediatric patients.
Genetic Targets Invigorate Research for Duchenne Muscular Dystrophy
February 22nd 2019Over the past several years, scientific teams have developed investigational methods for delivering a gene to correct a mutation in the DMD gene which causes DMD by creating dysfunction in a patient’s dystrophin production.
The Success of Risdiplam and Importance of Screening Infants for SMA
February 22nd 2019The professor of neurology and pediatrics at Columbia University Irving Medical Center spoke about the success of the small molecule in older infants with SMA, and about what she’s looking forward to in the future of SMA treatment.
Reldesemtiv Displays Promise Over Predecessor for ALS, Currently in Phase 2b Trial
February 20th 2019The investigational drug candidate, formerly known as CK-2127107, is the subject of the phase 2b FORTITUDE-ALS trial, which is assessing the effect of reldesemtiv compared with placebo on respiratory function and additional measures of skeletal muscle function.
FDA Issues Final Policy Framework for RMAT Designated Therapies
February 18th 2019The framework consists of 2 documents that expand on the agency’s plans for its risk-based approach for describing drugs, devices, and biologics, including those designated as regenerative medicine advanced therapies.
Rituximab Highly Efficacious in Myasthenia Gravis, Especially in MuSK Positive Patients
February 13th 2019At the final follow-up at an average of 20 months, remission was present in 42.9% of the 56 patients included, and an additional 25% had minimal manifestations or better as measured by the Myasthenia Gravis Foundation of America-Postintervention Status.
ALS Diagnostic Index Dependably Distinguishes ALS From Mimicking Disorders
February 9th 2019The study provided Class I evidence that the ALSDI can help the confirmation and diagnosis of ALS and its mimicking conditions at the earliest stages of the disease, when therapeutic efforts are most efficacious.
The Pipeline for Primary Mitochondrial Myopathy Finds Promise with Elamipretide
February 8th 2019The professor of pediatrics at Northeast Ohio Medical University and the director of the NeuroDevelopmental Science Center at Akron Children’s Hospital provided insight into the therapeutic landscape of PMM.